LP08 is a novel intravesical treatment for interstitial cystitis (IC), a poorly understood chronic disorder of unknown etiology consisting of irritative bladder symptoms and pelvic pain that dramatically affects quality of life. In IC/PBS, two distinct subtypes are generally recognized. Ulcerative (classic) IC and non-ulcerative IC/PBS clearly differ in terms of cystoscopic findings and symptom response to treatments. LP08 is a patent-protected investigational drug for IC comprised of pure liposomes. The purified phospholipid component of LP08 is known to be safe as a food source when eaten or delivered as a component of intravenous nutrition. There is no expectation that a novel route of bladder administration will present any safety issues. We will investigate the safety and efficacy of LP08 in an IND trial of ulcerative IC patients. Lipella Pharmaceuticals Inc. is a company formed by the inventors of LP08 in May of 2005. Lipella has received phase-I and phase-II SBIR support, as well as significant financial support from the Pittsburgh Life Sciences Greenhouse, a biotechnology investment organization, and accredited investors. The IND clinical trial will be conducted at the William Beaumont Hospital, the co-applicant of this STTR. Beaumont's urology department chairman, Kenneth Peters, is internationally recognized as a leader in the treatment and study of ulcerative IC and has a database of over 100 ulcerative IC patients. The urology department at the William Beaumont Hospital has been one of the leading centers in the world for the past 15 years on the study of IC and a top center in the NIDDK sponsored multi-institutional trials on IC including the ICDB, ICCTG and the ICCRN. This Fast-track STTR proposal advances commercial research funded by a prior phase-I NIH SBIR grant which has been completed, as well as a subsequent phase-II SBIR grant which has also been funded and is currently in progress. (The reason that the fast-track application process is chosen is so the pilot human batch produced in phase-I can produce both the regulatory data needed for the IND application and serve as the initial clinical supply without concerns for product shelf life and storage.) These prior and existing grants fund the preclinical development of a novel treatment for IC. The current STTR proposal would fund the clinical development of this product. Successful completion of this STTR would provide significant clinical data on a novel therapy for a syndrome with large unmet clinical demand, an asset highly attractive to institutional investors. In Summary, the aims of this proposal are designed to obtain significant clinical data for an experimental interstitial cystitis drug, by leveraging ongoing results of the cGMP liposome manufacturing capabilities at Lipella Pharmaceuticals and the IC-specific clinical capabilities of William Beaumont Hospital. PUBLIC HEALTH RELEVANCE: Successful completion of this STTR would provide significant clinical data on a novel therapy for interstitial cystitis, a syndrome with large unmet clinical demand. The development of an IND as well as the production of the first batch of product for human use is the focus of the phase-I portion of the current STTR proposal. IND approval from the FDA is the milestone between the phase-I and phase-II portions of this fast-track proposal. Manufacturing for and operational of the IND clinical trial is the focus of the phase-II portion of this STTR grant proposal.